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Monitor the compounds clinical development if it remains of interest
The Cranbury, New Jerseybased biotech announced FDA acceptance of its Biologics License Application for RP-L201 (marnetegragene autotemcel), an investigational lentiviral vector (LV)-based gene therapy for LAD-I, a rare, genetic immune disorder that predisposes patients to frequent and often fatal infections
Discontinuation due to adverse events was about 4% across all dose groups
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Limited, early-stage human data exist for this category
"Freeze-drying of proteins: process, formulation, and stability." Formulation and Delivery of Proteins and Peptides
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